Cystic Fibrosis
Conditions
Brief summary
The purpose of this study is to assess the pharmacokinetics and safety of VX-809 and VX-770 alone and in combination in healthy subjects.
Detailed description
This is a Phase 1, randomized, double-blind, placebo-controlled, multiple-dose study of orally administered VX-809 and VX-770 in healthy subjects. This study will evaluate safety and tolerability of VX-809 and VX-770 alone and in combination. Subjects will be randomized to receive study drug or placebo during three 14-day treatment periods separated by 14-day washout periods. In Treatment Period 1, subjects randomized to study drug will receive VX-809 every 24 hours. In Treatment Period 2, subjects randomized to study drug will receive VX-770 every 12 hours. In Treatment Period 3, subjects will receive VX-809 every 24 hours and VX-770 every 12 hours. Subjects randomized to placebo will receive placebo during all treatment periods. Enrollment is planned at 1 clinical site (Lenexa, Kansas). Up to 72 subjects will be enrolled. The study will be separated into 3 dose escalation cohorts. Cohort 1 and Cohort 2 will enroll 24 subjects. Cohort 3 is optional and may be conducted after a review of safety and pharmacokinetic data by Vertex.
Interventions
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* Male or female subject between 18 and 55 years of age, inclusive * Body mass index (BMI) between 18.5 and 30.0 kg/m2, inclusive * Subjects of child bearing potential and who are sexually active must meet the contraception requirements * Female subject must have a negative serum pregnancy test at screening and Day -1
Exclusion criteria
* History of any illness that, in the opinion of the investigator might confound the results of the study or pose an additional risk in administering study drug to the subject * Participated in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer) before the Screening visit * Subject who has received VX-770 or VX-809 in a previous clinical study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| PK parameters (including concentration, exposure and half-life) of VX-809 and its metabolite in plasma in the presence and absence of VX 770 | 70 days | Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-809 and its metabolite. |
| PK parameters (including concentration, exposure and half-life) of VX-770 and its metabolites in plasma in the presence and absence of VX 809 | 70 days | Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-770 and its metabolites. |
Secondary
| Measure | Time frame |
|---|---|
| Safety and tolerability as measured by adverse events (AEs) and clinically significant changes in laboratory values (clinical chemistry, hematology, coagulation, and urinalysis), electrocardiograms, and vital signs | 70 days |
Countries
United States
Outcome results
None listed